Gaucher Disease – Pipeline Review, H2 2019

Global Markets Direct’s, ‘Gaucher Disease – Pipeline Review, H2 2019’, provides an overview of the Gaucher Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Gaucher Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Gaucher Disease and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the global therapeutic landscape of Gaucher Disease

– The report reviews pipeline therapeutics for Gaucher Disease by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Gaucher Disease therapeutics and enlists all their major and minor projects

– The report assesses Gaucher Disease therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Gaucher Disease

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Gaucher Disease

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Gaucher Disease pipeline depth and focus of Indication therapeutics

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Adienne Pharma & Biotech

Apollo Therapeutics LLC

AVROBIO Inc

Belrose Pharma Inc

Bioasis Technologies Inc

Biosidus SA

Blue Turtle Bio Technologies Inc

Erad Therapeutics Inc

Evox Therapeutics Ltd

Freeline Therapeutics Ltd

Genzyme Corp

GT Gain Therapeutics SA

ISU ABXIS Co Ltd

Johnson & Johnson

Orphazyme A/S

Prevail Therapeutics Inc

Ranedis Pharmaceuticals LLC

Recursion Pharmaceuticals Inc

Sanofi

Takeda Pharmaceutical Co Ltd

Trucode Gene Repair Inc

Yuhan Corp

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Gaucher Disease Overview

Gaucher Disease Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Gaucher Disease Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Gaucher Disease Companies Involved in Therapeutics Development

Bioasis Technologies Inc

Genzyme Corp

JCR Pharmaceuticals Co Ltd

Johnson & Johnson

Lixte Biotechnology Holdings Inc

Orphazyme A/S

Gaucher Disease Drug Profiles

ADN-LYS Drug Profile

Product Description

Mechanism Of Action

R&D Progress

arimoclomol Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVRRD-02 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Biologic to Activate Glucocerebrosidase for Genetic Disorders Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Gaucher Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Gaucher Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ibiglustat Drug Profile

Product Description

Mechanism Of Action

R&D Progress

imiglucerase biosimilar Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LB-201 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LB-205 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LTI-291 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

miglustat Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NCGC-607 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Pcgin Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Protein for Type I and Type III Gaucher's Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RND-001 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate GBA for Gaucher Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

taliglucerase alfa biosimilar Drug Profile

Product Description

Mechanism Of Action

R&D Progress

xB-3007 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gaucher Disease Dormant Projects

Gaucher Disease Discontinued Products

Gaucher Disease Product Development Milestones

Featured News & Press Releases

Feb 06, 2018: Poster presentation at WORLDSymposium 2018 arimoclomol as clinical candidate for treatment of Gaucher disease

Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease

Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease

Jul 12, 2016: Researchers make advance in possible treatments for Gaucher, Parkinson's diseases

Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease

Nov 09, 2010: Actelion Receives Approval For Zavesca For Reimbursement Under Ontario Public Drug Program

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Gaucher Disease, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Products under Development by Universities/Institutes, H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Gaucher Disease – Pipeline by Adienne Pharma & Biotech, H2 2019

Gaucher Disease – Pipeline by Apollo Therapeutics LLC, H2 2019

Gaucher Disease – Pipeline by AVROBIO Inc, H2 2019

Gaucher Disease – Pipeline by Belrose Pharma Inc, H2 2019

Gaucher Disease – Pipeline by Bioasis Technologies Inc, H2 2019

Gaucher Disease – Pipeline by Biosidus SA, H2 2019

Gaucher Disease – Pipeline by Blue Turtle Bio Technologies Inc, H2 2019

Gaucher Disease – Pipeline by Erad Therapeutics Inc, H2 2019

Gaucher Disease – Pipeline by Evox Therapeutics Ltd, H2 2019

Gaucher Disease – Pipeline by Freeline Therapeutics Ltd, H2 2019

Gaucher Disease – Pipeline by Genzyme Corp, H2 2019

Gaucher Disease – Pipeline by GT Gain Therapeutics SA, H2 2019

Gaucher Disease – Pipeline by ISU ABXIS Co Ltd, H2 2019

Gaucher Disease – Pipeline by Johnson & Johnson, H2 2019

Gaucher Disease – Pipeline by Orphazyme A/S, H2 2019

Gaucher Disease – Pipeline by Prevail Therapeutics Inc, H2 2019

Gaucher Disease – Pipeline by Ranedis Pharmaceuticals LLC, H2 2019

Gaucher Disease – Pipeline by Recursion Pharmaceuticals Inc, H2 2019

Gaucher Disease – Pipeline by Sanofi, H2 2019

Gaucher Disease – Pipeline by Takeda Pharmaceutical Co Ltd, H2 2019

Gaucher Disease – Pipeline by Trucode Gene Repair Inc, H2 2019

Gaucher Disease – Pipeline by Yuhan Corp, H2 2019

Gaucher Disease – Dormant Projects, H2 2019

Gaucher Disease – Dormant Projects, H2 2019 (Contd..1), H2 2019

Gaucher Disease – Discontinued Products, H2 2019

List of Figures

List of Figures

Number of Products under Development for Gaucher Disease, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Number of Products by Targets, H2 2019

Number of Products by Stage and Targets, H2 2019

Number of Products by Mechanism of Actions, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019

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